PB2631: CLINICAL FEATURES, TREATMENTS AND OUTCOMES OF PRIMARY IMMUNE THROMBOCYTOPENIA IN ADULTS: A SINGLE CENTER RETROSPECTIVE STUDY FROM TUNISIA.
Authors: Yasmine Bnouni, Slama Nader, Sana Lakhel, Ons Charef, Nourhene Guedri, Sarra Boukhriss, Wiem Boufrikha, Zaineb Mlayah, Adnene Laatiri
Journal: HemaSphere (2023)

Topic: 32. Platelet disorders Background: Primary immune thrombocytopenia (ITP) is a rare autoimmune disease characterized by isolated peripheral thrombocytopenia (platelets<100 × 109/L). ITP is still considered as a diagnosis of exclusion which requires ruling out causes of secondary ITP. Aims: This study aims to report the epidemiological, clinical and therapeutic features of ITP in adults. Methods: We conducted a retrospective study including adult patients with ITP at the department of hematology at Fattouma Bourguiba Hospital in Tunisia over a period of 14 years (2008-2021). Results: A total of 78 patients was identified. The occurrence incidence was 5 new cases per year. The median age was 43 years old (18-82). The majority of patients were between 18 and 60 years old (79.5%) and only 20.5% of patients were > 60 years. The median age for women was 40 (18-82) and for men was 54 (18-80). Female predominance was noticed with a sex ratio of 0.32. This predominance decreased with age. In fact, the sex ratio was 0.22 for the age group [18-60] and 1 for patients > 60 years. ITP was discovered incidentally in 52.6% of cases and by bleeding in 39.7% of cases. By order of frequency, hemorrhagic manifestations were purpura, gingivorrhagia, menometrorrhagia, epistaxis and hematuria. Other symptoms such as asthenia and dizziness were found in 7.7% of cases. At diagnosis, the bleeding Khellaf score was high (> 8) in 16% of cases. The platelet count was ranging from 10× 109/L to 96 × 109/L (median of 36 × 109/L) and it was < 30 × 109/L in 55.8% of cases. Myelogram was performed in 51.3% of cases confirming the peripheral thrombocytopenia in all cases. Therapeutic abstention at diagnosis was indicated in 39.7% of cases and immediate treatment was required in 60,3% of cases. The mean duration of abstention was 27 months (3-96). Due to severe bleeding, eight patients (11%) were hospitalized and 5 patients (6.4%) needed platelet concentrate transfusion. Corticosteroid therapy was the first-line treatment in all our patients. Complete response (CR) was achieved in 54,3% of patients with newly diagnosed ITP, 25 % of patients with persistent ITP and in 43,3% of patients with chronic. The mean response time was 16 days (7-30) and the mean response duration was 28 months (1-144). Intravenous immunoglobulin associated with corticosteroids was prescribed for 22 patients with major bleeding (Khellaf score >8) with a CR in 8 patients. Other treatments were prescribed as second and third-line therapy in persistant and chronic ITP after corticosteroids. Two patients were treated by Rituximab with one partial response (PR) in 1 patient and failure in the other one. Danatrol was prescribed for 3 patients (1 obtained CR and 2 PR). One patient was treated by Azathioprine without response. Cyclophosphamide was prescribed for 2 patients (1 obtained CR and the other PR). Splenectomy was indicated to 7 patients after average of 42 months of diagnosis (CR for 3 patients). Eltrombopag was prescribed for 6 patients with a CR in all cases. Summary/Conclusion: ITP treatments are essentially based on corticosteroids. In case of cortico-dependence or cortico-resistance, other second and third-line treatments can be used such as rituximab, androgens, immunosuppressants and splenectomy. These treatments lack efficiency, hence the interest of thrombopoietin receptor agonists. Keywords: Corticosteroids, Immune thrombocytopenia (ITP)